This month we held a journalist briefing at the Science Media Centre, recruitment began for a new genetic therapy trial, our supporters continue to take on challenges for the Trust, and there was further coverage on the Creon supply issues.
Discover how Cystic Fibrosis Trust can support CF clinicians, enhance your career, and improve patient care with resources, support programmes, and training opportunities.
This week, we held a briefing for journalists in partnership with the Science Media Centre to mark the achievements that have been made in the last 60 years, whilst also looking to the future of CF and the Trust’s goal of everyone being able to live without limits imposed by CF.
When her daughter Phoebe was diagnosed with cystic fibrosis at seven months old, Rebecca found the support and information provided by Cystic Fibrosis Trust “invaluable” in the toughest of times. Here she shares Phoebe’s CF story and why the family won’t stop fundraising until there’s a cure.
This is a Phase 2, randomized, double-blind, placebo- and active-controlled, parallel group, multicenter study to evaluate the safety, tolerability, and efficacy of VX-440 in dual and triple combination with tezacaftor (TEZ; VX-661) and ivacaftor (IVA; VX-770) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F508del/F508del), or who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ and/or IVA therapy
This study will assess two emerging lung function tests. The main test uses lung MRI after inhaling a hyperpolarized gas (HP MRI), which images gas distribution within the lung. This test has been demonstrated in CF patients to show lung disease before other conventional test. So far this technique has been limited to small cohorts and therefore needs to be measured in larger cohorts. The second test is a breathing test entitled multiple breath washout (MBW). This test is similar to HP MRI and is also able to detect lung disease prior to conventional tests. It also measures gas distribution...
The study will evaluate a new hearing test - the High Frequency Digit Triplet (HFDT) test. This test mimics how individuals hear in real life situations (such as a noisy room), requires only a computer and does not need clinically trained personnel. We will evaluate the new test in stable patients and in those about to receive intravenous antibiotics for a chest infection. We will also see if it is feasible to do the test in primary school children. If the HFDT test is accurate and practical we will apply for a new research grant which would allow us to...
